Researchers identify potential for prenatal gene therapy

December 10, 2001

Minneapolis / St. Paul (December 10, 2001)--University of Minnesota (U of M) Cancer Center researchers will present findings that demonstrate promise for prenatal gene therapy to correct neurological and genetic disorders today (Monday, Dec. 10) at the American Society of Hematology Conference in Orlando, Fla.

Angela Panoskaltsis-Mortari, Ph.D., will show that the "Sleeping Beauty" transposon may be useful for in utero delivery of gene sequences capable of correcting congenital disorders of the blood and immune system that are challenging to treat post-natally.

"Sleeping Beauty" is an enzyme named by the U of M Cancer Center researchers who discovered its potential to link specific genes with specific functions (see Cell, November 1997). Mortari will demonstrate that when immune deficient mice fetuses were injected with the Sleeping Beauty enzyme, easily detectable green fluorescent protein (GFP) was seen in the liver, lung, heart, kidney, skin, and brain. Important for the correction of lymphohematopoietic genetic disorders, GFP expression was present in both the spleen and bone marrow, indicating that Sleeping Beauty can localize to hematopoietic organs in vivo.

Details of Mortari's presenation:

Time: Monday, December 10, 2001,
10:30 a.m. EST
Session name: Vector Development and Assessment
Title: In Utero Delivery of Sleeping Beauty Transposon Results in Long- Term Multi-Organ Expression: Potential for Prenatal Gene Therapy of Genetic Disorders.

Jakub Tolar, M.D., Ph.D., will show for the first time in mice that multipotent adult progenitor cells (MAPCs), or bone marrow stem cells, engraft and differentiate into brain and liver cells after in utero transfer. Their results support the possibility of future clinical use of adult bone marrow stem cells in attempts to correct neurological, hepatic, muscular and possibly other types of congenital disorders pre-natally.

Details of Tolar's presentation:

Time: Monday, December 10,
2001, 8:15 a.m. EST
Session name: Experimental Transplantation
Title: The in utero transfer of murine multipotent adult progenitor cells (MAPCs) results in brain and liver differentiation
Further details about their presentations can be found on the ASH Web site,

The University of Minnesota Cancer Center is a National Cancer Institute-designated Comprehensive Cancer Center. Awarded more than $68 million in peer-reviewed grants during fiscal year 2001, the center conducts cancer research that advances knowledge and enhances care. The center also engages community outreach and public education efforts addressing cancer. For more information on cancer in general, visit the Web site at or call 1-888-CANCER MN (1-888-226-2376 or 612/624-2620).


Sarah Youngerman, Academic Health Center, (612) 624-2346

Melanie Boulay, University of Minnesota Cancer Center, (612) 626-1107

University of Minnesota

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